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Gene Therapy at Nervous System

Medicine still has many challenges to solve specially on complex diseases were a large number of both, genetic and environmental factors are involved. Among them, neurodegenerative and autoimmune disorders affecting the central nervous system have attracted attention because CNS is difficult to access and to manipulate with classical pharmacological treatments and, in addition, there are no effective curative treatments for these diseases. To address these problems, we have focused our research interests in gene therapy strategies for autoimmune diseases and neurodegenerative disorders specially those associated to aging.
 

Team

Miguel Chillon Rodriguez

Miguel Chillon Rodriguez

Head of group
Gene Therapy at Nervous System
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Joan Roig Soriano

Joan Roig Soriano

Research technician
Gene Therapy at Nervous System
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Andrea Onieva Salgado

Andrea Onieva Salgado

Research technician
Gene Therapy at Nervous System
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Ángela Sánchez Osuna

Ángela Sánchez Osuna

Postdoctoral researcher
Gene Therapy at Nervous System
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Assumpció  Bosch  Merino

Assumpció Bosch Merino

Predoctoral researcher
Gene Therapy at Nervous System
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Beatriz Almolda Ardid

Beatriz Almolda Ardid

Postdoctoral researcher
Gene Therapy at Nervous System
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Miguel Chillon Rodriguez

Miguel Chillon Rodriguez

Head of group
Gene Therapy at Nervous System
Read more
Joan Roig Soriano

Joan Roig Soriano

Research technician
Gene Therapy at Nervous System
Read more
Andrea Onieva Salgado

Andrea Onieva Salgado

Research technician
Gene Therapy at Nervous System
Read more
Ángela Sánchez Osuna

Ángela Sánchez Osuna

Postdoctoral researcher
Gene Therapy at Nervous System
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Assumpció  Bosch  Merino

Assumpció Bosch Merino

Predoctoral researcher
Gene Therapy at Nervous System
Read more
Beatriz Almolda Ardid

Beatriz Almolda Ardid

Postdoctoral researcher
Gene Therapy at Nervous System
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Research lines

Gene Therapy Strategies for Esclerosis Multiple, an autoimmune disease affecting the nervous system

IP: -

Generation and development of more selective and less immunogenic viral vectors

IP: -

Molecular mechanisms involved in memory formation: Study of altered signaling pathways in CNS in non-pathogenic aging and in Alzheimer's Disease

IP: -

Projects

Terapia génica para MECP2 personalizada utilizando la tecnología CRISPR/Cas9 junto con administración por AAV, en cultivo de células 3D y ratones KI

IP: Miguel Chillon Rodriguez
Collaborators: -
Funding agency: Instituto de Salud Carlos III
Funding: 169097.5
Reference: AC20/00051
Duration: 01/01/2021 - 31/12/2023

Terapia génica para MECP2 personalizada utilizando la tecnología CRISPR/Cas9 junto con administración por AAV, en cultivo de células 3D y ratones KI

IP: Miguel Chillon Rodriguez
Collaborators: -
Funding agency: Instituto de Salud Carlos III
Funding: 169097.5
Reference: AC20/00051
Duration: 01/01/2021 - 31/12/2023

Recombinant HEBE: A chimeric Chronokine to rejuvenate the Brain

IP: Juan Francisco Espinosa Parrilla
Collaborators: -
Funding agency: Universitat Autònoma de Barcelona
Funding: 0.01
Reference: UAB/POC/2020
Duration: 12/03/2021 - 11/12/2021

Sol·licitud beques Predoctorals. Modalitat VHIR-AGAUR

IP: Miguel Chillon Rodriguez
Collaborators: Laia Perez Lasarte
Funding agency: Fundació Institut de Recerca HUVH
Funding: 46800
Reference: PRED-VHIR-16-34/EDO
Duration: 01/03/2017 - 29/02/2020

Sol·licitud beques Predoctorals. Modalitat VHIR-AGAUR

IP: Miguel Chillon Rodriguez
Collaborators: Laia Perez Lasarte
Funding agency: Fundació Institut de Recerca HUVH
Funding: 46800
Reference: PRED-VHIR-16-34/EDO
Duration: 01/03/2017 - 29/02/2020

Sol·licitud beques Predoctorals. Modalitat VHIR-AGAUR

IP: Miguel Chillon Rodriguez
Collaborators: Laia Perez Lasarte
Funding agency: Fundació Institut de Recerca HUVH
Funding: 46800
Reference: PRED-VHIR-16-34/EDO
Duration: 01/03/2017 - 29/02/2020

Estudio del papel de la isoforma soluble de alpha-Klotho en el inicio y progresión de déficits cognitivos: marcador de diagnóstico precoz, y molécula diana con potencial terapéutico

IP: Miguel Chillon Rodriguez
Collaborators: -
Funding agency: Instituto de Salud Carlos III
Funding: 165165
Reference: PI15/01270
Duration: 01/01/2016 - 31/12/2018

Sol·licitud beques Predoctorals. Modalitat VHIR-AGAUR

IP: Miguel Chillon Rodriguez
Collaborators: Laia Perez Lasarte
Funding agency: Fundació Institut de Recerca HUVH
Funding: 46800
Reference: PRED-VHIR-16-34/EDO
Duration: 01/03/2017 - 29/02/2020

Estudio del papel de la isoforma soluble de alpha-Klotho en el inicio y progresión de déficits cognitivos: marcador de diagnóstico precoz, y molécula diana con potencial terapéutico

IP: Miguel Chillon Rodriguez
Collaborators: -
Funding agency: Instituto de Salud Carlos III
Funding: 165165
Reference: PI15/01270
Duration: 01/01/2016 - 31/12/2018

Estudio del papel de la isoforma soluble de alpha-Klotho en el inicio y progresión de déficits cognitivos: marcador de diagnóstico precoz, y molécula diana con potencial terapéutico

IP: Miguel Chillon Rodriguez
Collaborators: -
Funding agency: Instituto de Salud Carlos III
Funding: 165165
Reference: PI15/01270
Duration: 01/01/2016 - 31/12/2018

Publications

AAV-mediated expression of secreted and transmembrane alphaKlotho isoforms rescues relevant aging hallmarks in senescent SAMP8 mice.

PMID: 35274439
Journal: AGING CELL
Year: 2022
Reference: Aging Cell. 2022 Apr;21(4):e13581. doi: 10.1111/acel.13581. Epub 2022 Mar 10.
Impact factor: 9.304
Publication type: Paper in international publication
Authors: Bosch, A, Pallas, M, Chillon, Miguel, Abraham, C R, Espinosa-Parrilla, J F, Roig-Soriano, J, Grinan-Ferre, C et al.
DOI: 10.1111/acel.13581

PROTEOMIC QUANTITATIVE STUDY OF DORSAL ROOT GANGLIA AND SCIATIC NERVE IN TYPE 2 DIABETIC MICE.

PMID: 34856394
Journal: Molecular Metabolism
Year: 2022
Reference: Mol Metab. 2022 Jan;55:101408. doi: 10.1016/j.molmet.2021.101408. Epub 2021 Nov 30.
Impact factor: 7.422
Publication type: Paper in international publication
Authors: Leal-Julia, Marc, Vilches, Jorge J, Onieva, Andrea, Verdes, Sergi, Sanchez, Angela, Chillon, Miguel, Navarro, Xavier, Bosch, Assumpcio et al.
DOI: 10.1016/j.molmet.2021.101408

A Transgenic Model Reveals the Role of Klotho in Pancreatic Cancer Development and Paves the Way for New Klotho-Based Therapy.

PMID: 34944918
Journal: Cancers
Year: 2021
Reference: Cancers (Basel). 2021 Dec 15;13(24). pii: cancers13246297. doi: 10.3390/cancers13246297.
Impact factor: 6.639
Publication type: Paper in international publication
Authors: Arbel Rubinstein, Tammi, Reuveni, Inbal, Hesin, Arkadi, Klein-Goldberg, Anat, Olauson, Hannes, Larsson, Tobias E, Abraham, Carmela R, Zeldich, Ella, Bosch, Assumpcio, Chillon, Miguel et al.
DOI: 10.3390/cancers13246297

Efficacy of AAV serotypes to target Schwann cells after intrathecal and intravenous delivery.

PMID: 34857831
Journal: Scientific Reports
Year: 2021
Reference: Sci Rep. 2021 Dec 2;11(1):23358. doi: 10.1038/s41598-021-02694-1.
Impact factor: 4.38
Publication type: Paper in international publication
Authors: Kagiava, A, Richter, J, Tryfonos, C, Leal-Julia, M, Sargiannidou, I, Christodoulou, C, Bosch, A, Kleopa, K A et al.
DOI: 10.1038/s41598-021-02694-1

PolyPurine Reverse Hoogsteen Hairpins Work as RNA Species for Gene Silencing.

PMID: 34576188
Journal: INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES
Year: 2021
Reference: Int J Mol Sci. 2021 Sep 16;22(18). pii: ijms221810025. doi: 10.3390/ijms221810025.
Impact factor: 5.924
Publication type: Paper in international publication
Authors: Aubets, Eva, Chillon, Miguel, Ciudad, Carlos J, Noe, Veronique et al.
DOI: 10.3390/ijms221810025

Specific Expression of Glial-Derived Neurotrophic Factor in Muscles as Gene Therapy Strategy for Amyotrophic Lateral Sclerosis.

PMID: 33786805
Journal: Neurotherapeutics
Year: 2021
Reference: Neurotherapeutics. 2021 Apr;18(2):1113-1126. doi: 10.1007/s13311-021-01025-6. Epub 2021 Mar 30.
Impact factor: 7.62
Publication type: Paper in international publication
Authors: Modol-Caballero, Guillem, Garcia-Lareu, Belen, Herrando-Grabulosa, Mireia, Verdes, Sergi, Lopez-Vales, Ruben, Pages, Gemma, Chillon, Miguel, Navarro, Xavier, Bosch, Assumpcio et al.
DOI: 10.1007/s13311-021-01025-6

Megalencephalic Leukoencephalopathy: Insights Into Pathophysiology and Perspectives for Therapy.

PMID: 33551753
Journal: Frontiers in Cellular Neuroscience
Year: 2020
Reference: Front Cell Neurosci. 2021 Jan 22;14:627887. doi: 10.3389/fncel.2020.627887. eCollection 2020.
Impact factor: 3.921
Publication type: Review in international publication
Authors: Bosch, Assumpcio, Estevez, Raul et al.
DOI: 10.3389/fncel.2020.627887

Cerebellar Astrocyte Transduction as Gene Therapy for Megalencephalic Leukoencephalopathy.

PMID: 32372403
Journal: Neurotherapeutics
Year: 2020
Reference: Neurotherapeutics. 2020 Oct;17(4):2041-2053. doi: 10.1007/s13311-020-00865-y.
Impact factor: 6.035
Publication type: Paper in international publication
Authors: Bosch, Assumpcio, Estevez, Raul, Nunes, Virginia, Sanchez, Angela, Garcia-Lareu, Belen, Puig, Meritxell, Prat, Esther, Ruberte, Jesus, Chillon, Miguel et al.
DOI: 10.1007/s13311-020-00865-y

Gene therapy for overexpressing Neuregulin 1 type I in skeletal muscles promotes functional improvement in the SOD1(G93A) ALS mice.

PMID: 32032731
Journal: NEUROBIOLOGY OF DISEASE
Year: 2020
Reference: Neurobiol Dis. 2020 Apr;137:104793. doi: 10.1016/j.nbd.2020.104793. Epub 2020 Feb 4.
Impact factor: 5.332
Publication type: Paper in international publication
Authors: Herrando-Grabulosa, Mireia, Garcia-Lareu, Belen, Solanes, Neus, Verdes, Sergi, Osta, Rosario, Francos-Quijorna, Isaac, Lopez-Vales, Ruben, Calvo, Ana Cristina, Bosch, Assumpcio, Navarro, Xavier et al.
DOI: 10.1016/j.nbd.2020.104793

Intrathecal AAVrh10 corrects Biochemical and Histological Hallmarks of Mucopolysaccharidosis VII Mice and Improves Behavior and Survival.

PMID: 31511867
Journal: HUMAN MOLECULAR GENETICS
Year: 2019
Reference: Hum Mol Genet. 2019 Nov 1;28(21):3610-3624. doi: 10.1093/hmg/ddz220.
Impact factor: 4.544
Publication type: Paper in international publication
Authors: Pages, G, Gimenez-Llort, L, Garcia-Lareu, B, Ariza, L, Navarro, M, Casas, C, Chillon, M, Bosch, A et al.
DOI: 10.1093/hmg/ddz220

Secreted alphaKlotho isoform protects against age-dependent memory deficits.

PMID: 29086766
Journal: MOLECULAR PSYCHIATRY
Year: 2018
Reference: Mol Psychiatry. 2018 Sep;23(9):1-11. doi: 10.1038/mp.2017.211. Epub 2017 Oct 31.
Impact factor: 13.204
Publication type: Paper in international publication
Authors: Masso, A, Sanchez, A, Bosch, A, Gimenez-Llort, L, Chillon, M et al.
DOI: 10.1038/mp.2017.211

Effects of Low Phytanic Acid-Concentrated DHA on Activated Microglial Cells: Comparison with a Standard Phytanic Acid-Concentrated DHA.

PMID: 29846873
Journal: NEUROMOLECULAR MEDICINE
Year: 2018
Reference: Neuromolecular Med. 2018 Sep;20(3):328-342. doi: 10.1007/s12017-018-8496-8. Epub 2018 May 30.
Impact factor: 2.952
Publication type: Paper in international publication
Authors: Ruiz-Roso, Maria Belen, Olivares-Alvaro, Elena, Quintela, Jose Carlos, Ballesteros, Sandra, Espinosa-Parrilla, Juan F, Ruiz-Roso, Baltasar, Lahera, Vicente, de Las Heras, Natalia, Martin-Fernandez, Beatriz et al.
DOI: 10.1007/s12017-018-8496-8

Soluble interleukin 23 receptor gene therapy with adeno-associated vectors for the treatment of multiple sclerosis.

PMID: 29171419
Journal: Neural Regeneration Research
Year: 2017
Reference: Neural Regen Res. 2017 Oct;12(10):1605-1606. doi: 10.4103/1673-5374.217327.
Impact factor: 1.769
Publication type: Editorail in international publication
Authors: Edo, Angel, Espinosa-Parrilla, Juan Francisco et al.
DOI: 10.4103/1673-5374.217327

Clinical and Histopathological Amelioration of Experimental Autoimmune Encephalomyelitis by AAV Vectors Expressing a Soluble Interleukin-23 Receptor.

PMID: 28593439
Journal: Neurotherapeutics
Year: 2017
Reference: Neurotherapeutics. 2017 Oct;14(4):1095-1106. doi: 10.1007/s13311-017-0545-8.
Impact factor: 5.166
Publication type: Paper in international publication
Authors: Eixarch, Herena, Tejero, Marcos, Costa, Carme, Hirota, Keiji, Castano, A Raul, Puig, Meritxell, Stockinger, Gitta, Montalban, Xavier, Bosch, Assumpcio, Espejo, Carmen et al.
DOI: 10.1007/s13311-017-0545-8

Development of a rapid, robust, and universal picogreen-based method to titer adeno-associated vectors.

PMID: 25640021
Journal: Human Gene Therapy Methods
Year: 2015
Reference: Hum Gene Ther Methods. 2015 Feb;26(1):35-42. doi: 10.1089/hgtb.2014.120.
Impact factor: 2.436
Publication type: Paper in international publication
Authors: Penalva, Cristina, Monfar, Merce, Chillon, Miguel, Miravet, Susana, Ontiveros, Maria, Piedra, Jose et al.
DOI: 10.1089/hgtb.2014.120

AAVrh.10 immunogenicity in mice and humans. Relevance of antibody cross-reactivity in human gene therapy.

PMID: 25410741
Journal: GENE THERAPY
Year: 2015
Reference: Gene Ther. 2015 Feb;22(2):196-201. doi: 10.1038/gt.2014.103. Epub 2014 Nov 20.
Impact factor: 3.104
Publication type: Paper in international publication
Authors: Thwaite, R, Pages, G, Chillon, M, Bosch, A et al.
DOI: 10.1038/gt.2014.103

Secreted and Transmembrane alphaKlotho Isoforms Have Different Spatio-Temporal Profiles in the Brain during Aging and Alzheimer's Disease Progression.

PMID: 26599613
Journal: PLoS One
Year: 2015
Reference: PLoS One. 2015 Nov 24;10(11):e0143623. doi: 10.1371/journal.pone.0143623. eCollection 2015.
Impact factor: 3.234
Publication type: Paper in international publication
Authors: Masso, Anna, Lizcano, Jose Miguel, Canete, Manuel, Garcia, Belen, Torres-Lista, Virginia, Puig, Meritxell, Bosch, Assumpcio, Chillon, Miguel, Gimenez-Llort, Lydia, Sanchez, Angela et al.
DOI: 10.1371/journal.pone.0143623

Mechanics of Viral Chromatin Reveals the Pressurization of Human Adenovirus.

PMID: 26491879
Journal: ACS Nano
Year: 2015
Reference: ACS Nano. 2015 Nov 24;9(11):10826-33. doi: 10.1021/acsnano.5b03417. Epub 2015 Oct 28.
Impact factor: 12.881
Publication type: Paper in international publication
Authors: Ortega-Esteban, Alvaro, Condezo, Gabriela N, Perez-Berna, Ana J, Chillon, Miguel, Flint, S Jane, Reguera, David, San Martin, Carmen, de Pablo, Pedro J et al.
DOI: 10.1021/acsnano.5b03417

Enhancing the Biological Relevance of Secretome-Based Proteomics by Linking Tumor Cell Proliferation and Protein Secretion.

PMID: 24897304
Journal: J PROTEOME RES
Year: 2014
Reference: J Proteome Res. 2014 Aug 1;13(8):3706-3721. doi: 10.1021/pr500304g. Epub 2014 Jul 15.
Impact factor: 5.001
Publication type: Paper in international publication
Authors: Gregori, Josep, Mendez, Olga, Katsila, Theodora, Pujals, Mireia, Salvans, Candida, Villarreal, Laura, Sanchez, Alex, Villanueva, Josep, Tabernero, Josep, Arribas, Joaquin et al.
DOI: 10.1021/pr500304g

Central nervous system delivery of helper-dependent canine adenovirus corrects neuropathology and behavior in mucopolysaccharidosis type VII mice.

PMID: 24299455
Journal: HUM GENE THER
Year: 2014
Reference: Hum Gene Ther. 2014 Mar;25(3):199-211. doi: 10.1089/hum.2013.152. Epub 2014 Jan 23.
Impact factor: 3.623
Publication type: Paper in international publication
Authors: Ariza, Lorena, Gimenez-Llort, Lydia, Pages, Gemma, Garcia-Lareu, Belen, Serratrice, Nicolas, Cots, Dan, Thwaite, Rosemary, Chillon, Miguel, Kremer, Eric J, Bosch, Assumpcio et al.
DOI: 10.1089/hum.2013.152

Experimental diabetes in neonatal mice induces early peripheral sensorimotor neuropathy.

PMID: 24846610
Journal: NEUROSCIENCE
Year: 2014
Reference: Neuroscience. 2014 Aug 22;274:250-9. doi: 10.1016/j.neuroscience.2014.05.015. Epub 2014 May 15.
Impact factor: 3.327
Publication type: Paper in international publication
Authors: Ariza, L, Pages, G, Garcia-Lareu, B, Cobianchi, S, Ruberte, J, Otaegui, P J, Chillon, M, Navarro, X, Bosch, A et al.
DOI: 10.1016/j.neuroscience.2014.05.015

Intrathecal administration of IGF-I by AAVrh10 improves sensory and motor deficits in a mouse model of diabetic neuropathy.

PMID: 26015946
Journal: Molecular therapy. Methods & clinical developm
Year: 2014
Reference: Mol Ther Methods Clin Dev. 2014 Jan 15;1:7. doi: 10.1038/mtm.2013.7. eCollection 2014.
Impact factor: 0
Publication type: Paper in international publication
Authors: Homs, Judit, Pages, Gemma, Ariza, Lorena, Casas, Caty, Chillon, Miguel, Navarro, Xavier, Bosch, Assumpcio et al.
DOI: 10.1038/mtm.2013.7

Functional characterization of the human mariner transposon Hsmar2.

PMID: 24039890
Journal: PLoS One
Year: 2013
Reference: PLoS One. 2013 Sep 11;8(9):e73227. doi: 10.1371/journal.pone.0073227. eCollection 2013.
Impact factor: 3.73
Publication type: Paper in international publication
Authors: Gil, Estel, Bosch, Assumpcio, Lampe, David, Lizcano, Jose M, Perales, Jose C, Danos, Olivier, Chillon, Miguel et al.
DOI: 10.1371/journal.pone.0073227

Helper dependent adenovirus vectors: progress and future prospects.

PMID: 24369061
Journal: CURRENT GENE THERAPY
Year: 2013
Reference: Curr Gene Ther. 2013 Oct;13(5):370-81.
Impact factor: 5.318
Publication type: Review in national publication
Authors: Cots, Dan, Bosch, Assumpcio, Chillon, Miguel et al.
DOI:

Short-fiber protein of ad40 confers enteric tropism and protection against acidic gastrointestinal conditions.

PMID: 23746215
Journal: Human Gene Therapy Methods
Year: 2013
Reference: Hum Gene Ther Methods. 2013 Aug;24(4):195-204. doi: 10.1089/hgtb.2012.096. Epub 2013 Jun 27.
Impact factor: 0
Publication type: Paper in international publication
Authors: Fernandez, Ester, Rodriguez, Ester, Romero, Carolina, Rio, Adolfo, Miralles, Marta, Raventos, Aida, Planells, Laura, Burgueno, Joan F, Hamada, Hirofumi, Perales, Jose Carlos et al.
DOI: 10.1089/hgtb.2012.096

Differentiated neuroprogenitor cells incubated with human or canine adenovirus, or lentiviral vectors have distinct transcriptome profiles.

PMID: 23922808
Journal: PLoS One
Year: 2013
Reference: PLoS One. 2013 Jul 26;8(7):e69808. doi: 10.1371/journal.pone.0069808. Print 2013.
Impact factor: 3.73
Publication type: Paper in international publication
Authors: Licursi, Valerio, Costa, Rossella, Roncaglia, Enrica, Gennetier, Aurelie, Ibanes, Sandy, Chillon, Miguel, Negri, Rodolfo, Tagliafico, Enrico, Kremer, Eric J, Saggio, Isabella et al.
DOI: 10.1371/journal.pone.0069808

Efficient amplification of chimeric adenovirus 5/40S vectors carrying the short fiber protein of Ad40 in suspension cell cultures.

PMID: 22860056
Journal: PLoS One
Year: 2012
Reference: PLoS One. 2012;7(7):e42073. doi: 10.1371/journal.pone.0042073. Epub 2012 Jul 31.
Impact factor: 4.092
Publication type: Paper in international publication
Authors: Miralles, Marta, Segura, Maria Mercedes, Puig, Meritxell, Bosch, Assumpcio, Chillon, Miguel et al.
DOI: 10.1371/journal.pone.0042073

Thesis

Generación de un adenovirus quimérico Ad5/52s pseudotipado con la proteína fiber corta del Ad52 para su caracterización in vitro e in vivo como vector de terapia génica

PhD student: Marc García Moure
Director/s: Miguel Chillon Rodriguez, Miguel Chillon Rodriguez
University: Universitat Autònoma de Barcelona
Year: 2016

Job offers

Laboratory Technician-Vector Production Unit, of the Group 'Teràpia Gènica per Sistema Nerviós'.
Start date:
22/11/2022
End date:
06/12/2022
Document: Download
Laboratory Technician-Gene Therapy Group for Nervous System.
Start date:
22/09/2022
End date:
06/10/2022
Document: Download
Laboratory Technician-Gene Therapy Group for Nervous System
Start date:
28/06/2022
End date:
12/07/2022
Document: Download

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